Multiple sclerosis (MS): Biotech bringing new impetus to research
Multiple sclerosis (MS) is a chronic, neurodegenerative disease that results in a decline in motor control and muscular strength in sufferers. In late stages, the disease leads to cognitive decline, loss of mobility, and eventual respiratory failure. The illness can be challenging to identify and presents in various forms that differ in aggression and severity.
The more progressive forms of the illness are sadly also those with the fewest available treatment options. Research from an array of companies is shining new light on potential therapeutic breakthroughs, however, which could reenergise the treatments market and at last bring a range of options to sufferers of progressive MS.
The more progressive forms of the illness are sadly also those with the fewest available treatment options. Research from an array of companies is shining new light on potential therapeutic breakthroughs, however, which could reenergise the treatments market and at last bring a range of options to sufferers of progressive MS.
Multiple Sclerosis
There are two main types of MS that can be broadly categorized as ‘relapsing’ or ‘non-relapsing’ courses of the disease. The first, relapsing-remitting (RRMS), advances more slowly, with flare-ups and remittance contributing to worsening and alleviated symptoms. Roughly 85 percent of diagnosis for MS are for RRMS. In this form, there are around 10 therapeutic treatments available, and MS sufferers can expect to live about 7 years fewer than non-sufferers, when using adequate treatments, albeit with disabilities.
The second form is primary progressive MS, which sees symptom development advancing more aggressively from the onset, without relapsing-remitting periods, resulting in more rapid neurodegenerative decline. Sufferers are likely to develop more severe symptoms and quality of life and life-expectancy can be further affected. At present, there are only a very limited number of therapeutic treatments on the market for this form of the illness.
Within 10 years, about 50 percent of RRMS cases will have transitioned to a more severe course of the disease, known as secondary progressive (SPMS), in which there is a gradual, continuous worsening of disability over time, and within 25 years about 90 percent of cases will have made this transition. Secondary progressive MS can be either ‘active’, with a relapsing pattern similar to RRMS and treated in much the same way, or ‘non active’, which is a disease course more closely resembling PPMS, without relapses and suffering from a similar dearth of treatment options.
An issue common to both ‘relapsing’ or ‘non-relapsing’ forms of MS is that modern treatments generally come with problems relating to immunosuppressive toxicity. This limits the viability of such treatments in prolonged therapies that exceed 3 years.
The second form is primary progressive MS, which sees symptom development advancing more aggressively from the onset, without relapsing-remitting periods, resulting in more rapid neurodegenerative decline. Sufferers are likely to develop more severe symptoms and quality of life and life-expectancy can be further affected. At present, there are only a very limited number of therapeutic treatments on the market for this form of the illness.
Within 10 years, about 50 percent of RRMS cases will have transitioned to a more severe course of the disease, known as secondary progressive (SPMS), in which there is a gradual, continuous worsening of disability over time, and within 25 years about 90 percent of cases will have made this transition. Secondary progressive MS can be either ‘active’, with a relapsing pattern similar to RRMS and treated in much the same way, or ‘non active’, which is a disease course more closely resembling PPMS, without relapses and suffering from a similar dearth of treatment options.
An issue common to both ‘relapsing’ or ‘non-relapsing’ forms of MS is that modern treatments generally come with problems relating to immunosuppressive toxicity. This limits the viability of such treatments in prolonged therapies that exceed 3 years.
The need is strong
In all its forms, MS is an extremely burdensome illness, both for sufferers and those close to them who must watch loved ones on a downward trajectory of disability. For all involved, there is a strong desire to see longer-lasting, less harmful treatments available. Associations and charities such as the Multiple Sclerosis Foundation and Myelin Repair Foundation, spread valuable information on MS and do important advocacy work, but the research that keeps their hopes afloat has not always produced viable treatment options.
There are roughly 2.5 million people living with MS, with 200 new cases diagnosed each week in the United States alone. Emerging therapies that have broad spectrums in terms of treatable pathologies have a distinct advantage in the market and are, therefore, more likely to make it to those who need them.
Presently, there is still a distinct limitation to the effectiveness of treatments for non-relapsing, progressive forms of MS. While sufferers of RRMS can expect to live a relatively long, mobile life, expectations for those who develop primary progressive MS are less heartening.
“One of the things I see regularly is patients complaining about not having a drug available to them for SPMS, at least in the United States,” said Ed Tobias, a senior patient columnist covering multiple sclerosis. “The focus of the pharmaceutical companies was, and seems to still be, the RRMS form of the disease,” he added.
Pricing is another big issue for sufferers of MS. The costs for treatment have increased dramatically, some sevenfold in the last 10 years. This puts many therapies at more $60,000 per year. With more products available hopefully this might encourage more competitive pricing, but requests for reductions remain unobserved by producers.
There are roughly 2.5 million people living with MS, with 200 new cases diagnosed each week in the United States alone. Emerging therapies that have broad spectrums in terms of treatable pathologies have a distinct advantage in the market and are, therefore, more likely to make it to those who need them.
Presently, there is still a distinct limitation to the effectiveness of treatments for non-relapsing, progressive forms of MS. While sufferers of RRMS can expect to live a relatively long, mobile life, expectations for those who develop primary progressive MS are less heartening.
“One of the things I see regularly is patients complaining about not having a drug available to them for SPMS, at least in the United States,” said Ed Tobias, a senior patient columnist covering multiple sclerosis. “The focus of the pharmaceutical companies was, and seems to still be, the RRMS form of the disease,” he added.
Pricing is another big issue for sufferers of MS. The costs for treatment have increased dramatically, some sevenfold in the last 10 years. This puts many therapies at more $60,000 per year. With more products available hopefully this might encourage more competitive pricing, but requests for reductions remain unobserved by producers.
Promising projects
Those concerned with progress in the fight against MS will be happy to learn that presently developments are emerging in many different areas of treatment, patient support, and diagnosis.
Simply diagnosing MS can be challenging and time-consuming. It is generally more of a process of eliminating other possible conditions than a clear search for specific symptoms. However, research indicates that the earlier the condition is identified the better the health outcomes for sufferers on average.
Through advances in MRI technology, researchers are helping to better define and recognise markers of MS onset, allowing doctors to catch the disease earlier through improved techniques of assessment. A group of researchers from the Berlin Institute of Health published findings in a study using an extremely powerful 7T MRI scanner.
Their findings supported the hypothesis that MS shares common pathogenetic mechanisms with another neurodegenerative disorder, but patients present with different types of neurological damage. This adds to a growing body of research regarding how to effectively identify MS.
Simply diagnosing MS can be challenging and time-consuming. It is generally more of a process of eliminating other possible conditions than a clear search for specific symptoms. However, research indicates that the earlier the condition is identified the better the health outcomes for sufferers on average.
Through advances in MRI technology, researchers are helping to better define and recognise markers of MS onset, allowing doctors to catch the disease earlier through improved techniques of assessment. A group of researchers from the Berlin Institute of Health published findings in a study using an extremely powerful 7T MRI scanner.
Their findings supported the hypothesis that MS shares common pathogenetic mechanisms with another neurodegenerative disorder, but patients present with different types of neurological damage. This adds to a growing body of research regarding how to effectively identify MS.
Molecule ALCAM
Researchers at the University of Montreal Hospital Research Center stopped the migration of immune B-cells through the blood-brain barrier of mice by blocking ALCAM, a molecule linked to the progression of multiple sclerosis (MS). This resulted in strong evidence that this could lessen disease severity in MS sufferers. While research is at an early stage, it bodes well for the next generation of MS therapies.
AB Science
One promising treatment is the compound masitinib, produced by AB Science. Being developed for a wide range of treatments in the areas of neurology, inflammatory disorders, and oncology, the compound is almost ready to be used by MS sufferers. This would be a crucial change to the treatments on offer for MS as evidence suggests masitinib could be a rare medication capable of treating both PPMS and non-active SPMS. Furthermore, long-term therapies of up to 10 years may well be possible.
The product also has applications for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, mastocytosis, severe asthma, prostate cancer and pancreatic cancer. To date, more than 6000 patients have been enrolled in masitinib studies, with strong data coming from the end of phase 3 trials.
The product also has applications for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, mastocytosis, severe asthma, prostate cancer and pancreatic cancer. To date, more than 6000 patients have been enrolled in masitinib studies, with strong data coming from the end of phase 3 trials.
Biogen
The multinational, neurological specialist has a strong portfolio of drug candidates at various stages of clinical trials. In particular, opicinumab demonstrated strong results in a phase 3 trial comparing it against Aubagio (teriflunomide) in the treatment of patients with relapsing MS.
AbbVie
The Abbot Laboratories spin-out is currently aiming to provide result on trials of elezanumab (ABT-555) at this year’s American Academy of Neurology (AAN).
Accord Healthcare
The provider of generic and specialty pharmaceuticals is conducting a phase 3 trial of simvastatin. The drug has shown positive signs in a phase 2 trial for both active and non-active secondary progressive MS, and the industry is eager to find out results when they are disclosed in 2023.
Companies